Orchard Therapeutics announces multiple presentations at ESGCT 2023

Orchard Therapeutics announced eight presentations from across its hematopoietic stem cell, HSC, gene therapy platform will be featured at the European Society of Gene and Cell Therapy, ESGCT, 30th Annual Congress taking place October 24-27, 2023, in Brussels. Featured presentations include a range of interim clinical outcomes in addition to the neurological and skeletal results previously reported from the company’s proof-of-concept study of OTL-203 in the Hurler subtype of mucopolysaccharidosis type I, as well as well as a first look at pre-clinical data demonstrating the use of vectorized HSCs as a delivery vehicle for monoclonal antibodies. In addition, several abstracts from the company’s pre-clinical research programs demonstrate the therapeutic potential of HSC gene therapy to address larger indications, including a genetic sub-type of frontotemporal dementia and Crohn’s disease, as well as chronic autoimmune disorders. “Together with our clinical collaborators, we’re proud of our significant presence at ESGCT which continues to underscore the transformative impact and broad applicability of our HSC gene therapy platform,” said Fulvio Mavilio, Ph.D., chief scientific officer of Orchard Therapeutics. “Collectively, these data demonstrate the potential of our approach to correct the underlying genetic defects responsible for several difficult-to-treat diseases, furthering our efforts toward unlocking the full promise of our portfolio.”